Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Neurology
3
Pipeline Programs
2
Companies
4
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
0
0
2
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

BridgeBio Pharma
BridgeBio PharmaCA - Palo Alto
2 programs
2
AG10Phase 31 trial
AcoramidisPhase 31 trial
Active Trials
NCT04418024Withdrawn0Est. Apr 2024
NCT04882735Withdrawn0Est. Oct 2026
Intellia Therapeutics
2 programs
1
NTLA-2001Phase 11 trial
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001N/A1 trial
Active Trials
NCT05697861Recruiting72Est. Mar 2038
NCT04601051Completed72Est. Sep 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
BridgeBio PharmaAcoramidis
BridgeBio PharmaAG10
Intellia TherapeuticsNTLA-2001
Intellia TherapeuticsLong-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Clinical Trials (4)

Total enrollment: 144 patients across 4 trials

NCT04882735BridgeBio PharmaAcoramidis

Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Start: Sep 2021Est. completion: Oct 20260
Phase 3Withdrawn
NCT04418024BridgeBio PharmaAG10

Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy

Start: Oct 2020Est. completion: Apr 20240
Phase 3Withdrawn
NCT04601051Intellia TherapeuticsNTLA-2001

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Start: Nov 2020Est. completion: Sep 202572 patients
Phase 1Completed
NCT05697861Intellia TherapeuticsLong-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Start: Jul 2023Est. completion: Mar 203872 patients
N/ARecruiting

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 144 patients
2 companies competing in this space

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